The proposed 21st Century Cures Act, which aims to promote the development of and access to new drugs, received overwhelming support by the House of Representatives, but experts have raised concerns about what certain provisions in the act could mean for managed care and health care.
"We have some concerns in terms of what its overall objectives are, and if you are talking about fine tuning a system to promote innovation and to create better medication, possibly even more affordable medications, I think that many of the provisions in the House's 21st Century Cures Act are ill-advised and won't have the intended result that many people are hoping," says Ameet Sarpatwari, JD, PhD, instructor in medicine at Harvard Medical School in the division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women's Hospital.
The House of Representatives passed the 21st Century Cures Act—a large piece of legislation with various components focusing on innovation—on July 10, 2015, before it was sent to the Senate later that month, but the Senate Health Committee announced earlier this year that instead of examining the act on the whole, they will likely address various aspects of the act separately.
"They will likely break it up into smaller pieces," says Mary Jo Carden, vice president of government and pharmacy affairs for the Academy of Managed Care Pharmacy (AMCP).
She says the Senate is expected to focus on individual aspects of the act such as additional funding for the National Institutes of Health (NIH) and the FDA. They are also anticipated to address aspects related to health information technology and privacy, as well as chronic conditions.
In addition, the US Senate is already making progress on efforts to curb the abuse of prescription drugs. On March 2, they approved an amendment to a larger prescription drug abuse bill that would give Medicare Part D the authority to require that beneficiaries at-risk for abuse would need to use a single prescriber and pharmacy for certain drugs. However, the overall bill will still need to be voted on by the Senate before it would move to the US House of Representatives.
Lowering the Bar
While it is still uncertain exactly how the Senate will choose to address various provisions within the 21st Century Cures Act or whether some provisions will be addressed at all, concerns have been raised about some of the aspects included within the proposed act.
For instance, Sarpatwari says he is particularly concerned about provisions related to medical devices and antimicrobials, or more specifically antibiotics, that could make it easier for these types of products to get approved and could ultimately result in more unsafe and ineffective drugs on the market driving up wasteful spending.
Under the 21st Century Cures Act, Sarpatwari says the FDA would be able to consider case histories and published peer-reviewed studies regardless of their quality when making decisions about the approval of high-risk medical devices. It would also create a breakthrough device pathway, which would give priority review to devices that are reportedly novel, similar to the breakthrough pathway already used for drugs.
But, Sarpatwari cautions that there are significant differences between the regulations for drugs and those for devices.
"First of all, the bar in terms of evidence that is sufficient to approve devices already is low comparative to drugs, and second, the ability to secure detailed and high-quality post-approval information, which would presumably be required for a lot of these products that are approved on the basis of surrogate endpoints, is more restrictive for devices," he says.
Sarpatwari says the FDA already has trouble forcing manufacturers to comply with post approval requirements and believes the provisions in the 21st Century Cures Act would create more problems.
Rita F. Redberg, MD, MSc, FACC, professor of medicine and core faculty at the Philip R Lee Institute for Health Policy Studies at the University of California, San Francisco (USCF) and director for the FAMRI Bland Lane Center for Excellence in Secondhand Smoke at USCF, agrees that provisions in the act would lower the standard for proving-safety and effectiveness of medical devices before they reach the market and are used in patients.
"Many high-risk devices are implanted, and cannot be safely removed if we later learn they are dangerous. There must be high-quality evidence to assure safety and effectiveness prior to use in patients. Case reports or expert opinion cannot assure safety and effectiveness of medical devices," she says.
Sarpatwari also questioned what devices would receive the breakthrough distinction.
"We've seen in the drug sphere the definition of what breakthrough means is very loosely interpreted and so you have follow-on products that are not necessarily that much better than existing products that are given classifications like this and so what that does is just speed up the introduction of products that aren't really going to help patient's necessarily," he says.
He also pointed to a proposal in the 21st Century Cures Act that he says would allow the approval of novel antimicrobials and anti-fungals based on preliminary, uncontrolled clinical trials with small sample sizes. Although these products would be given a disclaimer saying the product should be used in a limited population, Sarpatwari doubts its effectiveness.
"Evidence suggests that people don't really read those disclaimers," he says, adding that he believes it would potentially be putting unsafe and ineffective products into the market that might be using used for off-label indications.
Carden says while AMCP hasn't issued a strong statement to address these aspects of the 21st Century Cures Act, AMCP would want to ensure that drugs and biologics entering the market are safe.
"The FDA provides safe and effective medications and we don't want to lower the standard for doing that, so what's the right balance for getting products on the market that are safe and effective without having to compromise those issues particularly on the safety side," she says.
Overall, Sarpatwari says a common theme throughout the act is being faster.
"Do we need to be faster and what are the risks and benefits of being even faster in terms of safety and effectiveness?" he says. "Is that the type of innovation we want to bolster, because I don't think what this is doing—or I don't think these provisions—are really targeting the innovation we want to see."
Benefits to the System
While there are some concerns, there are also aspects of the act that are receiving positive praise from those in the health care industry.
For instance, the bill would provide an additional $8.75 billion to the NIH in fiscal years 2016-2020 through the creation of an Innovation Fund. In addition, $550 million would be given to the FDA over the next 5 fiscal years through the creation of a Cures Innovation Fund.
Carden says although AMCP would have liked to see the act focused more on funding the NIH and FDA, it is still a step in the right direction.
She says the FDA is "chronically underfunded." As a result, the industry is already seeing a backlog of biosimilars because of a lack of reviewers at the FDA to review the products. Currently, she says, it's also difficult for the FDA to hire scientists because of a significant difference in pay compared to private sectors.
"When there's a substantial difference in the private sector versus the government, particularly in those cases where you need a scientist that's very high level, very competent, etc, it's going to be very hard for the FDA to recruit," she says.
Sarpatwari also agrees that more funding would be a positive step for both the NIH and FDA but says politicians will also have to decide where that money would come from or if it's worth incurring more national debt.
"I would venture to say most reasonable people across both sides of the aisle who deal with this type of work would say that greater funding of FDA and NIH would be a good thing," he says.
Offering Guidance on Changes to FDAMA
The 21st Century Cures Act also had a provision that would re-examine language in the 1997 Food and Drug Administration Modernization Act (FDAMA) that was established to encourage manufacturers to share health care economic information with managed care pharmacy and therapeutic committees.
The Senate is expected to address the issue as well. AMCP believes the nearly 20-year-old law has been underutilized and held a partnership forum with various stakeholders in March to discuss changes they'd like to see.
According to a release distributed after the meeting, stakeholders agreed that dissemination of information should be expanded beyond health plan formulary committees to include organizations evaluating pharmacoeconomic information and others who are responsible for making coverage decisions in arrangements such as accountable care organizations.
“It’s essential that the potential of Section 114 finally be fully realized,” said AMCP CEO Susan A. Cantrell, RPh, CAE, in the release. “The extraordinary advances in medicines and rising concern over pharmaceutical costs make it imperative that health care decision makers have the information they need to evaluate the value of drugs and biologics.”
AMCP plans to use the feedback it gained from the meeting to draft guidance to the FDA and Congress.